A Phase 1/2 Study of VX-670 in Adult Participants With Myotonic Dystrophy 1 (DM1)
- Registration Number
- NCT06185764
- Lead Sponsor
- Vertex Pharmaceuticals Incorporated
- Brief Summary
The purpose of the study is to evaluate the safety, tolerability, pharmacokinetics, and pharmacodynamics of VX-670 at different single and multiple doses in participants with DM1.
- Detailed Description
Not available
Recruitment & Eligibility
- Status
- RECRUITING
- Sex
- All
- Target Recruitment
- 36
- Documented clinical diagnosis of DM1 with age of onset greater than (>) 1 year of age and documented positive genetic test for DM1 in the subject with cytosine thymine guanine (CTG) repeat of at least 100
Key
- History of any illness or any clinical condition as pre-specified in the protocol
Other protocol defined Inclusion/Exclusion criteria may apply.
Study & Design
- Study Type
- INTERVENTIONAL
- Study Design
- SEQUENTIAL
- Arm && Interventions
Group Intervention Description Part A: Single Ascending Dose VX-670 Participants will be randomized to receive a single dose of different dose levels of VX-670. Part A: Placebo Placebo Participants will be randomized to receive single dose of placebo matched to VX-670. Part B: Single and Multiple Ascending Dose VX-670 Participants will be randomized to receive single and multiple doses of different dose levels of VX-670. The dose levels will be determined based on the data from Part A. Part B: Placebo Placebo Participants will be randomized to receive single or multiple doses of placebo matched to VX-670.
- Primary Outcome Measures
Name Time Method Parts A and B: Safety and Tolerability as Assessed by Number of Participants With Adverse Events (AEs) Part A: From Baseline up to Day 42; Part B: From Baseline and up to Day 168
- Secondary Outcome Measures
Name Time Method Part B: Maximum Observed Concentration (Cmax) of VX-670 and its Active Component in Plasma After Each Dose From Day 1 up to Day 168 Part A: Area Under the Concentration Versus Time Curve (AUC) of VX-670 and its Active Component in Plasma From Day 1 up to Day 42 Part B: Area Under the Concentration Versus Time Curve (AUC) of VX-670 and its Active Component in Plasma After Each Dose From Day 1 up to Day 168 Part A: Maximum Observed Concentration (Cmax) of VX-670 and its Active Component in Plasma From Day 1 up to Day 42 Part B: Change in Splicing Index in Muscle Biopsy Baseline and at Day 15 Part B: Concentration of VX-670 and its Active Component in Muscle Baseline and at Day 15
Trial Locations
- Locations (26)
Universitaire Ziekenhuizen Leuven - Campus Gasthuisberg
🇧🇪Leuven, Belgium
Neuromuscular Reference Center Institute of Myology
🇫🇷Paris, France
Ludwig Maximilians Universitaet Muenchen
🇩🇪Muenchen, Germany
Centro Clinico NeMO
🇮🇹Milan, Italy
Hospital Universitario y Politécnico La Fe
🇪🇸Valencia, Spain
Leonard Wolfson Experimental Neurology Centre CRF
🇬🇧London, United Kingdom
Stanford Neuromuscular Research
🇺🇸San Carlos, California, United States
University of Florida Clinical Research Center
🇺🇸Gainesville, Florida, United States
University of Kansas Medical Center
🇺🇸Fairway, Kansas, United States
Boston Children's Hospital
🇺🇸Boston, Massachusetts, United States
Washington University School of Medicine / St. Louis Children's Hospital
🇺🇸Saint Louis, Missouri, United States
Wake Forest Baptist Health
🇺🇸Winston-Salem, North Carolina, United States
University of Pennsylvania
🇺🇸Philadelphia, Pennsylvania, United States
Virginia Commonwealth University (Sanger Hall)
🇺🇸Richmond, Virginia, United States
McGIll University
🇨🇦Montreal, Canada
Maastricht University Medical Center
🇳🇱Maastricht, Netherlands
Royal Hallamshire Hospital
🇬🇧Sheffield, United Kingdom
Wesley Research Institute
🇦🇺Auchenflower, Australia
Neuroscience Clinical Trials Unit, Alfred Brain
🇦🇺Melbourne, Australia
Hopital de Chicoutimi
🇨🇦Chicoutimi, Canada
Altasciences Montreal
🇨🇦Montreal, Canada
University of Ottawa
🇨🇦Ottawa, Canada
CHU Research Centre of Quebec
🇨🇦Quebec, Canada
Clinical Research Facility, Queen Elizabeth University Hospital
🇬🇧Glasgow, United Kingdom
St. George's University Hospital
🇬🇧London, United Kingdom
Salford Royal Hospital
🇬🇧Salford, United Kingdom